Study of Placebo or Bosentan to Treat Patients With Single Ventricle Physiology.

Summary

The purpose of this study is to determine whether Bosentan is an effective and safe treatment to adolescent and adult (15 years and older) patients, born with one ventricle of the heart instead of two (single ventricle physiology) and who have undergone TCPC as a palliative surgical treatment. The aim of the TCPC operation is to use the one functioning ventricle to pump the blood flow to the body, while the blood to the lungs is received directly from the caval veins, and is thus a passive flow, without the aid of a ventricle to actively pump the blood through the pulmonary circulation. The resistance in the pulmonary circulation is therefore critical to these patients. These patients have markedly lower work capacity in bicycle test than the general public. Furthermore they have a high risk of developing complications e.g. loss of protein from the intestines.

Bosentan is a medication that lowers the resistance in the pulmonary circulation. It is routinely used for patients with pulmonary hypertension. Some studies have shown that drugs that lower the pulmonary resistance can increase exercise capacity significantly in patients with single ventricle physiology.

In this study 80 patients will receive either placebo or Bosentan for 14 weeks. Before and after the treatment, bicycle test along with blood samples, stool samples and quality of life interviews will be performed. Every four weeks during the study blood samples, physical exam and interviews will be performed to ensure the safety of the treatment.

The investigators expect to find a significant increase in work capacity after 14 weeks in the treatment group compared with the placebo group.

Moreover the investigators hope to find a decrease in intestinal protein loss and an improved quality of life.

Conditions

• Hypoplastic Left Heart Syndrome
• Tricuspid Atresia
• Other Specified Congenital Anomalies of Heart

Interventions

DRUG

Bosentan

tablets Bosentan 62,5 mg x 2 daily for two weeks, then 125 mg x 2 daily for 12 weeks

DRUG

Placebo

Placebo tablets 2 x daily for 2 weeks, then change to different placebo tablets to match Bosentan group, 2 x daily for 12 weeks

Sponsors & Collaborators

• Aarhus University Hospital

  collaborator OTHER

• Bispebjerg Hospital

  collaborator OTHER

• Actelion

  collaborator INDUSTRY

• Rigshospitalet, Denmark

  lead OTHER

Principal Investigators

• Lars Sondergaard, DMSc · Rigshospitalet, Denmark

• Anders H Hebert, MD · Rigshospitalet, Denmark

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

15 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-02-28

Primary Completion

2013-03-31

Completion

2013-03-31

Countries

• Denmark
• Sweden

Study Locations